4D Molecular Therapeutics (FDMT) investor relations material

Strategic vision and market positioning

• Aims to reset the retinal disease treatment paradigm with a lifelong, always-on gene therapy, moving away from frequent bolus injections for wet AMD and DME.

• Sees a highly competitive and crowded market but believes its product profile is uniquely positioned for meaningful impact.

• Differentiates from other gene therapies by offering intravitreal delivery, which is easier to integrate into clinical practice compared to surgical or subretinal approaches.

• Expects its therapy to be considered for over 90% of AMD patients, except those well-controlled with minimal injections.

• Projects the global anti-VEGF market could reach $20 billion as the population ages, with significant opportunity for backbone therapy.

Clinical development and regulatory outlook

• Two pivotal Phase III studies for wet AMD are ongoing, with a pivotal DME trial planned to start in Q3, 2026.

• Regulatory alignment achieved with FDA, EMA, and Japan for a single global DME trial, in partnership with Otsuka for Asia-Pacific.

• Phase III trial designs are modeled after successful wet AMD studies, aiming for data that is both regulatory- and clinic-relevant.

• Non-inferiority trials use strict rescue criteria, designed in collaboration with retina experts to ensure clinical relevance.

• Upcoming catalysts include two-year PRISM follow-up data and DME SPECTRA trial updates in the second half of the year.

Commercial strategy and pricing

• U.S. is the initial launch priority, but global approval is targeted with trials designed for broad adoption.

• Partnership with Otsuka covers Asia-Pacific; European and other international markets are part of the long-term strategy.

• Pricing will be well below the million-dollar range typical for rare disease gene therapies, likely under $100,000.

• Manufacturing expertise and low dose requirements enable a cost of goods below $1,000, supporting competitive pricing.

• Large, established patient populations and ongoing incidence rates support a sustainable business model without rare disease pricing.